Pipeline Previews

Merck has announced that the FDA has designated lambrolizumab (MK-3475) as a Breakthrough Therapy for the treatment of patients with advanced melanoma. Lambrolizumab is Merck’s investigational antibody therapy targeting Programmed Death receptor (PD-1) that is currently being evaluated for the treatment of patients with advanced melanoma, and other tumor types.

The designation of an investigational drug as a Breakthrough Therapy is intended to expedite the development and review of a candidate that is planned for use, alone or in combination, to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

Lambrolizumab is an investigational antibody therapy designed to disrupt the action of the immune checkpoint protein PD-1 and therefore inhibit the ability of some cancers to evade the body’s immune system. Lambrolizumab is being studied in multiple cancer types including melanoma and non-small cell lung cancer.

Roche has announced that the European Union’s Committee for Medicinal Products for Human Use (CHMP) has recommended conditional approval of Erivedge (vismodegib) for the treatment of adult patients with symptomatic metastatic basal cell carcinoma, or locally advanced basal cell carcinoma inappropriate for surgery or radiotherapy. Conditional approval would make Erivedge the first licensed treatment for patients in Europe with these disfiguring, debilitating and even fatal forms of skin cancer, which are known collectively as advanced basal cell carcinoma.

The CHMP stated, on the basis of quality, safety and efficacy data submitted, considers there to be a favorable benefit-to-risk balance for Erivedge and therefore recommended the granting of the marketing authorization. This marketing authorization is conditional, and will require the submission of additional data from ongoing studies. The CHMP grants conditional approval to medicinal products that fulfill an unmet medical need.

The conditional recommendation was based on findings from the ERIVANCE BCC phase II study which enrolled 104 advanced basal cell carcinoma patients (71 had locally advanced and 33 had metastatic disease) from 31 study sites in the US, Australia and Europe. The study showed Erivedge substantially shrank tumors or repaired visible lesions, as defined by objective response rate, in 42.9 percent of patients with locally advanced and 30.3 percent of patients with metastatic basal cell carcinoma as assessed by independent review.

The most common adverse events included muscle spasms, hair loss, altered taste sensation, fatigue and weight loss. Serious adverse events (SAEs) were observed in 26 patients (25 percent), however of these only four (4 percent) patients had SAEs that were considered to be related to treatment with Erivedge. Fatal events were reported in seven patients (7 percent) although none were considered by investigators to be related to treatment with Erivedge. In all cases, patients had other pre-existing diseases or symptoms that were related to their presumed cause of death.

The safety profile of Erivedge is being further assessed in STEVIE, a global, single-arm, open-label, multicentre study of patients with advanced forms of basal cell carcinoma. The study aims to enroll 1,200 patients. An interim analysis from STEVIE confirmed a similar safety profile to that observed in the ERIVANCE BCC study.

Erivedge is a Hedgehog pathway inhibitor, designed to selectively target abnormal signaling in a cell growth pathway, known as the Hedgehog signaling pathway. The Hedgehog signaling pathway is implicated in the development of certain types of cancer, including basal cell carcinoma.

The FDA approved Erivedge in 2012 under the priority review program that provides for an expedited six-month review of drugs that offer major advances in treatment, making it the first licensed medicine for patients with advanced basal cell carcinoma.

Taro Pharmaceutical Industries Ltd. has reported that it received approval from the FDA for its New Drug Application Topicort^®(desoximetasone) Topical Spray, 0.25%.

Topicort^® (desoximetasone) Topical Spray, 0.25% is a corticosteroid indicated for the treatment of plaque psoriasis in patients 18 years of age or older.

The FDA has announced that it has approved Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G)-(Equine) to treat patients showing signs of botulism following documented or suspected exposure to botulinum neurotoxin. The product is derived from horse plasma and contains a mixture of antibody fragments that neutralize all of the seven botulinum nerve toxin serotypes known to cause botulism.